10 Participants Needed

FMT for Autism

RP
SM
Overseen BySonia Michail, MD
Age: < 18
Sex: Any
Trial Phase: Phase 1
Sponsor: Children's Hospital Los Angeles

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether participants need to stop taking their current medications. It's best to discuss this with the trial coordinators or your doctor.

How does the treatment FMT for autism differ from other treatments?

FMT (Fecal Microbiota Transplantation) is unique because it involves transferring gut bacteria from a healthy donor to a patient, which is different from other autism treatments that typically focus on cognitive or behavioral therapies. This approach is novel as it targets the gut-brain connection, which is not a standard focus in autism treatments.12345

What is the purpose of this trial?

The purpose of this study is to find out if transplant of fecal matter (stool), also known as fecal microbiota transplantation (FMT), from a healthy person into the intestines of children and young adults with Autism Spectrum Disorder (ASD).For this study children between the ages of 5-17years will be recruited over 2 years. Children will be recruited who receive an ASD diagnosis using the gold-standard Autism Diagnosis Observation Schedule -2 (ADOS-2) using module 1, 2 or 3 (none, limited or no moderate expressive language). Children diagnosed with these modules of the ADOS-2 may be at greater risk for GI disorders and rigid-compulsive behaviors. Additional assessment of rigid-compulsive behaviors and social communication will be done using the Repetitive Behavioral Scales-Revised (RBS-R) and Social Responsiveness Scale-2 (SRS-2), respectively. KBIT (the Kaufman Brief Intelligence Test) is used at baseline to obtain patient IQ. Total evaluation time is approximately 90 minutes. Following baseline symptom evaluation, a medical exam will be performed to determine whether each child is expressing specific GI symptoms. In addition, parents will fill out the Questionnaire for Pediatric Gastrointestinal Symptoms- Rome III (QPGS-III). Once an ASD diagnosis is confirmed, FMT treatment will be initiated, which typically occurs within 4-6 weeks of the initial diagnosis. Half 50% of the children (n=5) will receive the equivalent of 50 g of stools from a healthy donor into the jejunum through upper endoscopy and the other 50% off children (n=5) will receive Saline solution as Placebo control through upper endoscopy.Subjects will have a total of 5 visits within 24 weeks including phone call follow up on Day 7 after FMT.

Research Team

SM

Sonia Michail, MD

Principal Investigator

Children's Hospital Los Angeles

PL

Pat Levitt, Ph.D

Principal Investigator

Children's Hospital Los Angeles

Eligibility Criteria

This trial is for children aged 5-17 with Autism Spectrum Disorder (ASD) who have gastrointestinal symptoms. They must be diagnosed using specific autism and GI symptom assessments, need an upper GI endoscopy, and not have any severe sensory or motor problems, genetic syndromic disorders, significant renal/liver dysfunction, immunodeficiency conditions, or severe food allergies.

Inclusion Criteria

ADOS validated diagnoses of ASD
Questionnaires: RBS-2, KBIT, SRS, Rome III Version (QPGS- RIII), Ped QL, SSP
I am between 5 and 17 years old and have been diagnosed with autism.
See 2 more

Exclusion Criteria

You were born before 36 weeks of pregnancy.
Pregnancy: testing will be done on FMT day 0 for subjects with childbearing potential
I am capable of giving consent but choose not to.
See 4 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

4-6 weeks
1 visit (in-person)

Baseline Evaluation

Baseline symptom evaluation and medical exam to determine GI symptoms, including assessments using ADOS-2, RBS-R, SRS-2, and KBIT

Approximately 90 minutes
1 visit (in-person)

Treatment

Participants receive FMT or placebo through upper endoscopy

1 day
1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including a phone call follow-up on Day 7

24 weeks
5 visits (including phone call follow-up)

Treatment Details

Interventions

  • FMT
Trial Overview The study tests if fecal microbiota transplantation (FMT)—transferring stool from a healthy donor to the intestines—helps kids with ASD. Participants are randomly chosen to receive either FMT or a saline placebo through an endoscopy into the jejunum.
Participant Groups
2Treatment groups
Active Control
Placebo Group
Group I: Arm 2Active Control1 Intervention
Active Comparator: Donor Stool Transplant Arm 2 will get FMT (Fecal Microbial Transplant) with Healthy Donor Stool into the jejunum through upper endoscopy.
Group II: Arm 1Placebo Group1 Intervention
Placebo Comparator placebo into the jejunum through upper endoscopy.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Children's Hospital Los Angeles

Lead Sponsor

Trials
257
Recruited
5,075,000+

Findings from Research

Cognitive Remediation Therapy (CRT) significantly improved executive functions and school performance in children and adolescents with Autism Spectrum Disorder (ASD), with 16 out of 25 participants completing an average of 22 sessions.
After CRT, participants showed notable enhancements in intellectual abilities, working memory, and specific aspects of cognitive flexibility, indicating that CRT is a promising intervention for addressing cognitive impairments associated with ASD.
Cognitive remediation therapy in autism spectrum disorder: Tunisian experience.Hajri, M., Abbes, Z., Ben Yahia, H., et al.[2020]

References

Cognitive remediation therapy in autism spectrum disorder: Tunisian experience. [2020]
The anatomy of the callosal and visual-association pathways in high-functioning autism: a DTI tractography study. [2021]
Emotional Rational Education Training Associated with Mindfulness for Managing Anxiety within Adolescents Affected by High-Functioning Autism: A Descriptive Study. [2021]
Age of Speech Onset in Autism Relates to Structural Connectivity in the Language Network. [2021]
Infantile autism: adult outcome. [2019]
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